Gene Therapy Trial for CLN6 Batten Disease

Starting soon · Phase 1/Phase 2

Conditions studied: CLN6, Batten Disease, Batten's Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis

In brief

The goal of this clinical trial is to learn if a gene therapy called scAAV9.CB.CLN6 can treat children with CLN6 Batten disease (variant late infantile neuronal ceroid lipofuscinosis). The main questions it aims to answer are if he gene therapy safe and well tolerated, and if the gene therapy help slow disease progression or improve symptoms. Participants will: Receive a single dose of the gene therapy through an injection into the fluid around the spinal cord (intrathecal administration) Have regular study visits over 2 years for safety checks and assessments of disease progression Be followed for an additional 3 years in a long-term follow-up study

Key facts

Study ID

NCT07582484

Run by

The Charlotte and Gwenyth Gray Foundation

People needed

12

Starts

2026-08-01

Expected to finish

2028-08-01

Last updated by the study team

2026-05-14

Who can join

Age: 0 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of CLN6
• At least 4 months old

You may not qualify if…

• Presence of another inherited neurologic disease
• Prior stem cell transplantation
• Prior gene transfer, gene editing, or viral vector therapy

Where it is running

• University of California, San Diego - Rady Children's — La Jolla, California, United States

Full record on ClinicalTrials.gov

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